
By Michael Erman
NEW YORK (Reuters) -The U.S. Food and Drug Administration can approve new personalized treatments for rare and deadly genetic diseases based on data from a handful of patients, two of the agency's top officials said on Wednesday.
FDA Commissioner Marty Makary and Chief Medical and Scientific Officer Vinay Prasad said in an essay published in the New England Journal of Medicine that for certain conditions, companies could rely on appropriately designed studies with small sample sizes rather than randomized trials. They will rely on biological plausibility and clinical improvements in those early patients.
"Current regulations are onerous and unnecessarily demanding," Makary and Prasad wrote. "For patients and families, there is no time to wait."
The new "plausible-mechanism" pathway would allow the agency to grant marketing authorization after manufacturers demonstrate success with several consecutive patients.
Companies that receive these approvals will be required to collect real-world evidence to confirm efficacy continues and to look for safety issues that might arise.
The new approach will prioritize treatments for rare diseases that are fatal or cause severe childhood disability. Common diseases with unmet medical needs may also qualify.
While makers of cell and gene therapies are likely to be significant beneficiaries of the new approval process, Makary and Prasad said that other types of treatments could also receive licensure this way.
"The FDA will work as a partner and guide in ushering these therapies to market," the officials wrote.
(Reporting by Michael ErmanEditing by Bill Berkrot)
latest_posts
- 1
Top 15 Supportable Design Brands Coming out on top - 2
Artemis II's moon-bound astronauts capture Earth's brilliant blue beauty as they leave it behind - 3
'Women on the floor, riddled with bullets': Ex-hostage Rom Braslavski recounts 'horrors' of Oct. 7 - 4
Scientists uncover an ant assassination scheme that helps a parasitic queen rise to power - 5
IDF struck Iran's largest petrochemical plant, second facility hit in two days, Katz confirms
Feeling wonder every day improves our health. Here’s how to do it.
Survey: Canteen Cups With Great Warm Protection Impact
Kristin Cavallari was the teen queen bee of 'Laguna Beach.' Now she's a 'cringey' mom.
‘RuPaul's Drag Race’ Season 18: How to watch without cable, premiere time, cast list and more
How did life begin on Earth? New experiments support 'RNA world' hypothesis
Figure out How to Explore Land Close to 5G Pinnacles
Which European palace do you fantasy about visiting? Vote!
4 Masked Men Steal Renoir, Matisse and Cézanne Paintings Worth Over $10 Million in 3-Minute Heist
2 ways you can conserve the water used to make your food













